How will Novartis price its groundbreaking CAR-T med? R&D exec offers some clues

Novartis has an FDA action date on CAR-T drug CTL019 in early October.

The FDA isn't the only hurdle for Novartis' forthcoming CAR-T therapy, which could be the world's first. There's pricing and access, too. Drugs in this new class of one-time treatments are expected to be ultra-expensive, so the company is working up pricing and payment strategies now.

Novartis’ CTL019 is under review to treat pediatric and young adult relapsed and refractory B-cell acute lymphoblastic leukemia (ALL), with the FDA’s action date set for early October. In the meantime, it's using stem cell transplants as a benchmark for pricing and considering pay-for-performance deals to make the medications accessible for payers and patients alike, R&D chief Vas Narasimhan told Bloomberg.

CAR-T therapies are individualized treatments manufactured from a patient's own T cells, with the promise of a cure. As one-time treatments, just as gene therapies are, they're expected to be among the world's most costly drugs if approved. Novartis is in a hot race to approval with Kite Pharma, with other would-be CAR-T products coming up behind.

Earlier this year, England’s cost watchdogs NICE ran the numbers and found that CAR-T medications could be worth up to $649,000, assuming that patients gain 10 quality-adjusted life years over the current standard of care. At the time, a Novartis spokesperson said "it is too early for us to comment on pricing; however, we will at the time of approval."

Asked to confirm the Narasimhan's remarks on pricing and payment, a Novartis spokesperson reiterated that it’s “too early for us to comment on pricing.” Stem cell transplants and follow-up treatment can cost upwards of $800,000.

Narasimhan’s statements come just days after an investor and analyst meeting for Novartis, where the company’s management talked up CAR-T prospects. In their coverage of the meeting, Jefferies analysts wrote that they “detected a strong change in tone and optimism around the CAR-T program.”

In a note afterward, Jefferies analyst Jeffrey Holford wrote that Novartis management was “enthusiastic about the ability to price CAR-T at very attractive levels as well as to also potentially move into the [chronic lymphocytic leukemia] indication, multiple myeloma and solid tumors.” Those indications would be big expansions, potentially adding thousands more patients eligible for treatment.

Novartis plans a filing in diffuse large B-cell lymphoma this year, according to the pipeline report on its website. On Wednesday, the drugmaker released interim findings in relapsing/remitting diffuse large B-cell lymphoma patients showing a 3-month overall response rate in 45% of the patients. The med hit its primary endpoint.

An April ranking of the world’s costliest meds determined that uniQure’s gene therapy Glybera topped the industry at $1.2 million, but low demand forced the company to pull the plug on that med since the numbers were compiled. Recent pricey launches include Biogen’s spinal muscular atrophy med Spinraza, which costs $750,000 for the first year and half that for subsequent years, and BioMarin’s Brineura to treat the ultrarare CLN2 disease, at $702,000.

Novartis is vying against Kite Pharma, Juno and others in the groundbreaking CAR-T field. Kite’s axicabtagene ciloleucel is due for an FDA action by Nov. 29 in refractory non-Hodgkin's lymphoma, according to BioPharmCatalyst. EvaluatePharma analysts have pegged the Kite drug, KTE-C19, as one of the top 10 drug launches of 2017.